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What treatment with PALYNZIQ looks like

PALYNZIQ® (pegvaliase-pqpz) Injection is a daily at-home injectable treatment.

Here are a few things to know about how you will take PALYNZIQ:

  • You will give yourself daily injections just under the skin (subcutaneously) while at home
  • PALYNZIQ comes in prefilled syringes that can be injected in a few steps

 

PALYNZIQ is available in 3 dosage strengths:

 

Image of single dose syringes for palynziq

A needle smaller than a flu shot

You should know a few key differences between the PALYNZIQ needle and the other needles you may be familiar with. PALYNZIQ uses a short, thin needle that:

  • Is smaller than most vaccines you may have received (like a flu shot)
  • Requires only the space of a pinch of skin in a soft, fatty body area
  • Retracts automatically after completing the injection

Hesitant about self-injection?* Learn about the educational and support options available to you>

*PALYNZIQ uses a subcutaneous injection, which is inserted into the fatty tissue just under the layer of skin. It does not go as far into the body as an intramuscular injection.

Finding a dose to meet your treatment goals

Your clinic team will slowly adjust the dose of your PALYNZIQ to find a dose that works for you.

You will start by injecting PALYNZIQ once a week. Then, your healthcare provider will adjust your dose based on how you’re responding to therapy.

Here is an example of how dosing with PALYNZIQ may look:

Dosing Chart
palynziq dosing chart

†Additional time may be required prior to each dosage escalation based on patient tolerability.

‡Individualize treatment to the lowest effective and tolerated dosage. Consider increasing to 40 mg once daily in patients who have not achieved a response with 20 mg once daily continuous treatment for at least 24 weeks. Consider increasing to a maximum of 60 mg once daily in patients who have not achieved a response with 40 mg once daily continuous treatment for at least 16 weeks.

§PALYNZIQ should be discontinued if patients have not achieved an adequate response after 16 weeks of continuous treatment at the maximum dosage of 60 mg once daily.

Everyone responds differently to PALYNZIQ and finding the right dose for you will take time.

The reason PALYNZIQ is introduced slowly and over time is to allow your immune system to get used to the medication.

Videos to help with your PALYNZIQ journey

Play

A step-by-step guide on how to inject PALYNZIQ

Learn about injecting and administering PALYNZIQ with this step-by-step video.

Play

Overcoming the fear of needles

Get more comfortable with needles and injections with a brief overview of the product, education, and support that you’ll receive when taking PALYNZIQ.

Play

Real Talk: Self-injections

Watch adults with PKU share how their PALYNZIQ injection fits into their daily schedules.

Before injecting PALYNZIQ, talk to your healthcare provider right away if you cannot or will not use auto-injectable epinephrine to treat a severe allergic reaction. If you are pregnant or plan to become pregnant while taking PALYNZIQ, talk to your healthcare provider to discuss the risks and benefits of taking PALYNZIQ during pregnancy to you and your unborn baby. If you are breastfeeding or plan to breastfeed, talk to your healthcare provider to discuss the risks and benefits of taking PALYNZIQ while breastfeeding for you and your baby.

You’re not alone—when you start taking PALYNZIQ, your clinic team will:

  • Show you how to properly inject PALYNZIQ
  • Be present as you give yourself your first injection
  • Confirm your ability to self-administer PALYNZIQ
  • Teach you about the signs and symptoms of anaphylaxis, and what to do if you have anaphylaxis
  • Instruct you on how to use auto-injectable epinephrine in case it’s needed due to anaphylaxis
    • You must carry your auto-injectable epinephrine with you at all times

Side effects can occur with PALYNZIQ

PALYNZIQ can cause a severe allergic reaction (anaphylaxis) that may be life threatening and can happen at any time during treatment with PALYNZIQ.

Your clinic team will train you to recognize the signs and symptoms so you’ll be ready and well-prepared to manage this reaction, should it occur. These include:

  • Fainting (passing out)
  • Dizziness or lightheadedness
  • Sudden confusion
  • Trouble breathing or wheezing
  • Chest discomfort or chest tightness
  • Fast heart rate
  • Swelling of your face, lips, eyes, or tongue
  • Throat tightness
  • Flushed skin
  • Skin rash, itching, or raised bumps on skin
  • Nausea, vomiting, or diarrhea
  • Losing control of urine or stools

10% of patients (29 of 285) experienced anaphylaxis in clinical studies

  • Of the 10% of patients who experienced anaphylaxis in PALYNZIQ studies, 72% of them were able to return to treatment with PALYNZIQ under the supervision of their clinic team
  • Of those patients who returned to treatment, the majority (71%) did not experience anaphylaxis again
  • All anaphylaxis episodes in the study ended without further health consequences for the patients
For your safety, your clinic team will prescribe auto-injectable epinephrine. Keep the auto-injectable epinephrine with you at all times during treatment with PALYNZIQ.

Common side effects when patients in clinical studies were starting (induction) and increasing (titration) their PALYNZIQ dose (N=285)

Your clinic team will start you on a low dose and slowly increase your dose so that your immune system can adjust to PALYNZIQ.

Graphic about palynziq side-effects
  • While most patients in clinical studies experienced a side effect while taking PALYNZIQ, only 15% of people had to stop treatment due to side effects
  • During maintenance (ongoing), common side effects were similar to those seen when starting therapy and while increasing therapy
  • It’s also important to know that while most patients in clinical studies experienced a side effect while taking PALYNZIQ, only 15% of people stopped treatment due to side effects
Most side effects seen with PALYNZIQ in clinical studies were considered mild to moderate and generally decreased over time—many of which can be managed.

View a video about anaphylaxis

Play

Anaphylaxis explained

This video will help you learn more about the potential risk of anaphylaxis with PALYNZIQ and how your care team will ensure you know how to respond if it ever were to occur.

The PALYNZIQ Risk Evaluation and Mitigation Strategy (REMS)

You can get PALYNZIQ only through the PALYNZIQ REMS program. The purpose of the program is to ensure that you have been fully informed of the risk of anaphylaxis associated with taking PALYNZIQ, and that you understand this risk before you start treatment. Your clinic team can tell you more about PALYNZIQ REMS and help you enroll.

Your journey with PALYNZIQ

If you and your clinic team decide that PALYNZIQ® (pegvaliase-pqpz) Injection is right for you, here are the steps you will follow to get started:

Step 1

Discuss PALYNZIQ with your clinic team.

Your healthcare provider will take you through the PALYNZIQ Risk Evaluation and Mitigation Strategy (REMS), which will help you understand:

  • The risks associated with PALYNZIQ, including a severe allergic reaction (anaphylaxis)
  • The need for auto-injectable epinephrine

Afterwards, you’ll fill out a patient authorization form that allows BioMarin RareConnections™ to contact you about support services.

Step 2

Get support throughout your treatment journey.

In person or over the phone, BioMarin Clinical Coordinators will work closely with you to provide injection training and ongoing reinforcement of how to properly inject PALYNZIQ. They will also provide ongoing product education and support throughout your treatment journey.

Step 3

Learn more from a BioMarin RareConnections™ Case Manager.

They will work with your clinic team and support you by confirming insurance benefits, identifying financial assistance options, and coordinating shipment of medication. Your Case Manager will provide information about financial assistance options, including the PALYNZIQ Co-Pay Assistance Program for eligible patients.

Step 4

Receive PALYNZIQ in the mail.

PALYNZIQ is only available from pharmacies that participate in the PALYNZIQ REMS.

The pharmacy will:

  • Verify that you have received and filled a prescription for auto-injectable epinephrine
  • Call you to schedule a shipment of PALYNZIQ that will come right to your home

Step 5

Administer your first injection under the supervision of a healthcare professional.

A trained healthcare professional will be there to teach you how to inject PALYNZIQ, answer any questions, help you with dosage adjustments, and monitor your blood Phe levels.

Step 6

Stay on therapy with the help of ongoing support.

BioMarin is committed to providing personalized product education and support services to help you start and stay on therapy. You’ll have access to comprehensive resources and individualized support throughout your treatment with PALYNZIQ.

biomarin logo

BioMarin Clinical Coordinators are available to support you throughout your treatment journey. In person or over the phone, they will work closely with you to provide injection training and ongoing reinforcement of how to properly inject PALYNZIQ.

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IMPORTANT SAFETY INFORMATION

What is the most important information I should know about PALYNZIQ?

PALYNZIQ can cause a severe allergic reaction (anaphylaxis) that may be life threatening and can happen any time during treatment with PALYNZIQ.

Severe allergic reactions are a serious but common side effect of PALYNZIQ.

  • You will receive your first injection of PALYNZIQ in a healthcare setting where you will be closely watched for at least 1 hour after your injection for a severe allergic reaction
  • Your healthcare provider will prescribe auto-injectable epinephrine for you, and will teach you (or your caregiver) and your observer, if needed, when and how to use it if you have a severe allergic reaction
  • If you have a severe allergic reaction during treatment with PALYNZIQ, you will need to receive an injection of epinephrine immediately and get emergency medical help right away
  • Your healthcare provider will decide if you (or your caregiver) are able to give the PALYNZIQ injections, recognize the signs and symptoms of a severe allergic reaction, give an injection of epinephrine, and call for emergency help, if needed
  • Your healthcare provider may recommend that an adult observer (or your caregiver) be with you when you give your PALYNZIQ injection and for at least 1 hour after your injection to watch you for signs and symptoms of a severe allergic reaction and, if needed, give you an injection of epinephrine and call for emergency medical help

Stop injecting PALYNZIQ and get emergency medical care right away if you have any of the following symptoms:

  • Fainting (passing out)
  • Dizziness or lightheadedness
  • Sudden confusion
  • Trouble breathing or wheezing
  • Chest discomfort or chest tightness
  • Fast heart rate
  • Swelling of your face, lips, eyes, or tongue
  • Throat tightness
  • Flushed skin
  • Skin rash, itching, or raised bumps on skin
  • Nausea, vomiting, or diarrhea
  • Losing control of urine or stools

Keep the auto-injectable epinephrine with you at all times during treatment with PALYNZIQ. Read the Patient Information that comes with the auto-injectable epinephrine that your healthcare provider prescribes for you for more information.

If you have a severe allergic reaction, do not continue to take PALYNZIQ until you talk with your healthcare provider. Your healthcare provider will tell you if you can continue treatment with PALYNZIQ.

People taking PALYNZIQ have also experienced allergic reactions other than anaphylaxis. Talk to your healthcare provider if you experience any allergic reactions when taking PALYNZIQ.

PALYNZIQ REMS: PALYNZIQ is available only through a restricted program called the PALYNZIQ REMS (Risk Evaluation and Mitigation Strategy). Talk to your healthcare provider about the PALYNZIQ REMS and how to enroll.

What should I tell my healthcare provider BEFORE starting PALYNZIQ?

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Before injecting PALYNZIQ, talk to your healthcare provider right away if you cannot or will not use auto-injectable epinephrine to treat a severe allergic reaction. If you are pregnant or plan to become pregnant while taking PALYNZIQ, talk to your healthcare provider to discuss the risks and benefits of taking PALYNZIQ during pregnancy to you and your unborn baby. If you are breastfeeding or plan to breastfeed, talk to your healthcare provider about the best way to feed your baby if you take PALYNZIQ.

Before injecting PALYNZIQ, read the Medication Guide and Instructions for Use that come with your PALYNZIQ injection.

What should I watch for AFTER starting PALYNZIQ?

PALYNZIQ may cause serious side effects, including:

  • Severe allergic reactions (anaphylaxis)
  • Other allergic reactions to PALYNZIQ can happen during treatment with PALYNZIQ. Contact your healthcare provider right away if you have any of the following symptoms of an allergic reaction including: rash, itching, or swelling of the face, lips, eyes, or tongue. Your healthcare provider may change your dose of PALYNZIQ, stop your treatment with PALYNZIQ for a period of time, or prescribe medicine for you to take before your PALYNZIQ injection to help reduce the symptoms of an allergic reaction

The most common side effects of PALYNZIQ include injection site reactions (such as redness, itching, pain, bruising, rash, swelling, or tenderness), joint pain, headache, skin reactions that spread and last at least 14 days (such as itching, rash, or redness), nausea, stomach pain, vomiting, cough, mouth and throat pain, itching, diarrhea, stuffy nose, feeling very tired, dizziness, anxiety, and low levels of Phe in your blood.

These are not all of the possible side effects of PALYNZIQ. Speak with your healthcare provider right away about any side effects.

Important notes

Blood Phe testing and diet

  • Your healthcare provider will monitor your blood Phe levels during PALYNZIQ treatment
  • Monitor the amount of protein and Phe that you eat or drink. Your healthcare provider may change the amount of protein and Phe you should have in your diet during treatment with PALYNZIQ, depending on the levels of Phe in your blood. Follow your healthcare provider’s instructions about the amount of protein and Phe you should have in your diet

Missed dose

  • If a dose is missed, take your next dose as scheduled and do not take 2 doses of PALYNZIQ to make up for the missed dose

Pregnancy Surveillance Program

  • There is a pregnancy surveillance program for females who take PALYNZIQ during pregnancy, or who become pregnant while receiving PALYNZIQ or within 1 month after their last dose of PALYNZIQ. The purpose of this program is to collect information about the health of you and your baby while taking PALYNZIQ. Talk to your healthcare provider about how you can take part in this program or call BioMarin at 1-866-906-6100

You may report side effects to BioMarin at 1-866-906-6100.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please see full Prescribing Information, including an important warning for risk of anaphylaxis, and the Medication Guide.

US-PAL-00519 02/25

What is the most important information I should know about PALYNZIQ?

PALYNZIQ can cause a severe allergic reaction (anaphylaxis) that may be life threatening and can happen any time during treatment with PALYNZIQ.

What is PALYNZIQ?
PALYNZIQ® (Pal-lin-zeek) (pegvaliase-pqpz) is a prescription medication used to lower blood levels of phenylalanine (Phe) in adults with PKU (phenylketonuria) who have uncontrolled blood Phe levels above 600 micromol/L (10 mg/dL) on their current treatment. You should discuss the potential benefits and risks of PALYNZIQ with your healthcare provider.